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Safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A

WHAT IS THE PURPOSE OF THIS STUDY?

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

Study Identifiers

IS THIS STUDY RIGHT FOR ME?

AGE

UP TO 5

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

ENTRY CRITERIA

Inclusion Criteria1. Participant is < 6 years old at the time of screening2. Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening3. Participant has severe hemophilia A (Factor VIII (FVIII) < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A4. Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by the central laboratory at screening5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocolAdditional inclusion criteria for Part B (immune tolerance induction (ITI))1. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion2. Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with a. poorly controlled bleeding despite increased BAX 855 doses, or b. requires bypassing agents to treat bleeding

Exclusion Criteria1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening2. Participant has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease)4. Participant has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for ≥ 3 EDs at any time prior to screening5. Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion.6. The participant’s weight is anticipated to be < 5 kg at the baseline visit7. Participant’s platelet count is < 100,000/mL8. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 809. Participant has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented international normalized ratio (INR) > 1.5] in his medical history or at the time of screening10. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)11. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation12. Participant is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral chemotherapy13. Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study14. Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance15. Parent, legally authorized representative or participant are a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.Additional exclusion criteria for Part B (ITI)1. Spontaneous disappearance of the inhibitor prior to ITI2. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory3. Inability or unwillingness to comply with the protocol

Study Locations

We are planning* on conducting this study in 22 countries around the world.

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Important information regarding the use of this website

You are about to download Clinical Study Report (CSR) synopses or factual lay summaries on clinical trials for medicines/indications approved in the US and EU on or after January 1, 2015.
Access to these documents requires that you acknowledge and accept the following:
• The purpose of this website is to increase transparency regarding the outcome of clinical trials sponsored by Shire and is strictly non-promotional. It is not intended to promote any prescription, sale or use, whether approved or off-label, of any Shire medicinal products. This website is for informational purposes only and is not intended to have any legally binding effect.
• Members of the public having any questions regarding the use of any of the products referred to in these documents should consult a healthcare professional for additional information and medical advice. They should not rely on the information from this website to draw any conclusions about healthcare products, treatments or treatment options.
• The information provided on this website is not intended to be used as prescribing information. Please refer to applicable authority-approved labeling information for your country.
• The documents on this website are provided in the same format in which they were originally prepared for submission to regulatory authorities for marketing approval; exceptions include redactions necessary for protection of personal data and commercially confidential information.
• The information contained in these documents represents the results known at the time of the study’s completion. Shire does not accept any liability for the accuracy, completeness or utility of this information. There is no obligation in connection with updating the documents and information available on this website. Your access and use of this website and the content it contains is at your own risk.

Important information regarding the use of this website

You are about to download Clinical Study Report (CSR) synopses or factual lay summaries on clinical trials for medicines/indications approved in the US and EU on or after January 1, 2015.
Access to these documents requires that you acknowledge and accept the following:
• The purpose of this website is to increase transparency regarding the outcome of clinical trials sponsored by Shire and is strictly non-promotional. It is not intended to promote any prescription, sale or use, whether approved or off-label, of any Shire medicinal products. This website is for informational purposes only and is not intended to have any legally binding effect.
• Members of the public having any questions regarding the use of any of the products referred to in these documents should consult a healthcare professional for additional information and medical advice. They should not rely on the information from this website to draw any conclusions about healthcare products, treatments or treatment options.
• The information provided on this website is not intended to be used as prescribing information. Please refer to applicable authority-approved labeling information for your country.
• The documents on this website are provided in the same format in which they were originally prepared for submission to regulatory authorities for marketing approval; exceptions include redactions necessary for protection of personal data and commercially confidential information.
• The information contained in these documents represents the results known at the time of the study’s completion. Shire does not accept any liability for the accuracy, completeness or utility of this information. There is no obligation in connection with updating the documents and information available on this website. Your access and use of this website and the content it contains is at your own risk.